The Cytel blog keeps you up to speed with the latest developments in biostatistics and clinical biometrics.

Avoiding Lost-in-Translation with Submission Terminology

February 24, 2021

In a previous post, I discussed the importance of proper use of CDISC Controlled Terminology (CDISC CT) in SDTM. However, the CDISC-CT is not the only submission terminology you need to be familiar with when building SDTM datasets to be submitted to the FDA (and similarly to the PMDA). As per the FDA Data Standards Catalog, when submitting datasets to the agency, you need to follow not only the CDISC standards (SDTM, ADAM, define-xml and CDISC-CT) but also a number of other submission terminologies. For example, this is the case of MedDRA when your SDTM package contains Adverse Events data, or WHO Drug Dictionary for Medications, but there are also a number of other submission terminologies you need to apply, particularly in the TS – Trial Summary Dataset.

A number of presentations and papers have been published discussing TS domain[1] and clarify requirements that are not always fully clear in the SDTM IG or in the agencies Technical Conformance Guide.

In this blog, I focus on TS and discuss some specific parameters that you need to submit in TS using various “external” dictionaries, and help you understand how to find the correct term (and code).

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A little walk in the CDISC Library, hand in hand with SAS

January 28, 2021

The Christmas break presented an opportunity to make my first concrete steps into the CDISC Library. Overall, it was a pleasant “promenade”.

The CDISC Library forms the foundation of an ongoing transformation in the way we will access and make use of the CDISC standards to facilitate the long awaited and desired, end-to-end data process implementation (see also the CDISC 360 project).

With the availability of the CIDSC Library, vendors can now develop software which you can use to instantly access standards i.e., Standards Controlled Terminology or data standards (for example SDTM). Standards are now available in machine readable and non-proprietary format.

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Submitting Software Programs to the Regulatory Agencies, what should you know?

December 18, 2020

Can I submit software programs other than SAS? What software programs should I submit? Are sponsors required to submit executable programs?

Do I need to rename my software programs so that they all have the same extension e.g. “.txt”?

Can I make use of macros in my software programs and if so, should macros be part of the submission package?

What kind of documentations for software programs should I include in the submission package?

Do I need to follow any particular style and conventions when writing software programs that will be part of a submission package?

A single topic generates so many questions! Get the answers in this blog.

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Data Standards and Submission Highlights from PHUSE EU CONNECT 2020

November 23, 2020

The Virtual PHUSE-EU CONNECT Conference was held from November 8 to 13 and the event was a great success, despite all of us missing the face-to-face contact.

The conference kicked-off on Sunday night with a Social Virtual event with a “Numerologist Show”. Of course this could not replace and compete with the usual “toast” we were used to do live, so we did it virtually (check out my LinkedIn post where I offer some cocktails recommendations and share the recipe of my favorite cocktail “The Negroni” with a bit of history. But please don’t do it before Friday night, you will need the weekend to recover).

Like every year, Cytel significantly contributed to the event as one of the official sponsors, running a workshop (“Predictive Analytics Using R”), chairing and co-chairing two streams (Machine Learning & Connected Health and Scripts and Macros), preparing four on demand presentations (in Application and Development, Coding and Tricks and Data Standards and Governance streams) and two posters.

In this blog, I focus on presentations related to data standards and data submission to agency, in general.

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Staffing Needs for RWE Delivery

November 3, 2020

When an expert statistician is paired with an experienced set of data managers, opportunities to capitalize on quantitative strategy are spotted more quickly. Statisticians can determine whether datasets can strengthen study findings by being presented in a way that uses the available data in a scientifically objective way that is at the same time in line with the clients’ strategic objectives.

The practice of combining statistical needs with the processes of data management and other related services for real world evidence, we will henceforth call RWE-Delivery. There are several models for RWE-delivery that can similarly vary with the needs of a study. Questions about process, management and timelines are just as key for this choice of delivery model, as the objectives of the delivery. Therefore, it is important to work closely with delivery teams to determine the possible needs for study completion.

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The Missing Link: Risking your Traceability (and “Credibility”) when your ADaM package is not traceable back to SDTM

October 28, 2020

About three years ago, Cytel was helping a sponsor on a project where I had to conduct surveillance of some CRO deliverables, mainly for SDTM and ADaM packages. At first, I was involved in the review cycle of SDTM, and began by reviewing some initial mapping specifications including a draft SDTM Annotated CRF. The CRO in charge was quite experienced and there was nothing major to spot in all the different versions I had to review.

Surprisingly, it was not the case some months later, when I had to provide the same review support for the Biostatistics deliverables, specific to the ADaM package. The ADaM datasets overall were well designed, and there were no major open non-conformance issues. However, it was clear from the very beginning that there was something missing - a missing link between SDTM and ADaM.

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Interview with Thomas Wilke: Health Economics and Real-World Evidence Studies

October 20, 2020

In this interview with Thomas Wilke, Principal Scientist at Ingress-Health (a Cytel company), we talk to him about his background and experience in Health Economics, understand the important considerations of real-world evidence studies and the impact of COVID-19 pandemic on the work of the health economics outcomes researchers who work at Ingress and Cytel. We also cover important HEOR topics such as its benefits for market access studies and real-world analytics (RWA) for regulatory submission.

Cytel and Ingress-Health will be contributing to a range of events at Virtual ISPOR EU 2020, on November 16th – November 19th. Our Real-World analytics teams will be collaborating to deliver a number of interactive workshops, issue panels, posters and podiums to showcase their work and share innovative insights in HEOR, evidence generation, knowledge synthesis and decision analysis.

Click below to download our full list of sessions at ISPOR EU

download the  sessions schedule

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An Interview with Bart Heeg (Part 1):The Increasing Importance of Health Economics

October 14, 2020

A credible evidence base is needed to support and document the economic value of new technologies and therapeutic approaches. Companies need careful cost-effectiveness analyses for successful reimbursement submissions. In this two-part blog series, we interview Bart Heeg, Vice President HEOR and Founder at Ingress Health (A Cytel company). Bart talks about his background in HEOR, founding Ingress Health and its recent acquisition by Cytel. He also talks about the benefits of turning to an HEOR specialist and provides a sneak peek into Cytel’s presentations at the upcoming ISPOR EU 2020 event.

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Introducing Observational Studies – Three Trends for Statisticians

October 13, 2020

The combination of greater access to electronic health records, bigger electronic claims datasets, and the need for more clinical insight in ensuring patient safety, has made observational studies an important new tool in trial design. Observational studies typically take non-randomized data from outside of a trial and use quantitative and modeling techniques to draw conclusions from big datasets. While typically used for HEOR and market access, augmenting regulatory submissions with observational studies is gaining prominence. As with all data analyses, there is an implicit rule of ‘garbage in-garbage out,’ where data that is not up to the standard required for the formation of sound scientific judgment, should not be used. Sponsors should rely on the most sophisticated tools and advanced analytics to make the most rigorous use of available data.

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